SarcoidosisPhase 4NCT02920710
ACTHAR Therapy for Central Nervous System Sarcoidosis
A Phase 4 study testing Repository Corticotropin Injection in people with Sarcoidosis, run by The Cleveland Clinic at 1 site in United States.
Trial summary
There is a need for a more reliable, expeditious therapy that can be used as an alternative to glucocorticoids in severe Central Nervous System (CNS) sarcoidosis. This study aims to provide evidence for effectiveness of ACTHAR gel in CNS sarcoidosis, and provide information about its safety and tolerability Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Sarcoidosis
- What is being tested?
- Repository Corticotropin Injection
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 0 participants
- Where
- 1 siteUnited States
- Study dates
- 1 Feb 2019to 2 Nov 2020
- Phase
- Phase 4
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Any age
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Patient with sarcoidosis as defined by ATS/ERS/WASOG (American Thoracic Society/European Thoracic Society/World Association for Sarcoidosis and Other Granulomatous Disorders)
- Stable baseline immunosuppressive medications
- Moderate to severe disease as defined by at least one of the following criteria:
- Cranial nerve palsy
- Neurologic deficits related to intraparenchymal brain, spinal cord and/or cauda equina involvement
- Dural or leptomeningeal involvement of brain and/or spinal cord
- Hydrocephalus
- Seizures
Who cannot join
- Diagnosis of any underlying neurologic disorder that would potentially confound interpretation of the study results
- Significant change in corticosteroid dose within the past 4 weeks, or other immunosuppressive medication within the past 6 months
- Evidence of current serious infection, or a history of chronic or recurring infections.
- Contraindication to high-dose corticosteroids (e.g. uncontrolled blood sugar).
- Allergies to pig-derived proteins
- Have a history of any opportunistic infection within 6 months prior to screening
- History of malignancy.
Trial locations and contacts
1 site in 1 country.
About this study
There is a need for a more reliable, expeditious therapy that can be used as an alternative to glucocorticoids in severe Central Nervous System (CNS) sarcoidosis. This study aims to provide evidence for effectiveness of ACTHAR gel in CNS sarcoidosis, and provide information about its safety and tolerability
Read the detailed description
Central nervous system (CNS) involvement is one of the most severe manifestations of sarcoidosis. Sarcoidosis affecting the leptomeninges, spinal cord, or brain parenchyma portends a difficult course and frequently results in severe disability or death (1). Treatment of moderate and severe CNS sarcoidosis typically involves a combination of corticosteroids and cytotoxic agents such as methotrexate (2). Unfortunately, most response rates are reportedly only in the 29-38% range for corticosteroids alone, and the effects of cytotoxic agents in sarcoidosis require up to 6 months to occur. A typical scenario is that patients are treated for prolonged periods with high dose glucocorticoids with suboptimal effectiveness despite development of substantial toxicities. Some series report that cyclophosphamide or infliximab may be beneficial (3), but these approaches are limited by potentially severe toxicities, loss of effectiveness, or payor constraints.
. ACTHAR is a 39-amino acid peptide natural form of adrenocorticotropin hormone (ACTH) that was initially approved in 1952 by the FDA. It has since been approved for 19 indications including respiratory sarcoidosis, multiple sclerosis, and infantile spasms.
- Conditions
- Sarcoidosis
- Being tested
- Repository Corticotropin Injection
- Medical subject headings
- Sarcoidosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Repository Corticotropin InjectionDrug
Also called H.P. Acthar Gel, ACTH Gel, ACTH
- Initial treatment with 80 units daily for ten days (induction phase)
- Maintenance treatment with 80 units twice weekly (maintenance phase)
| Study group | Role | What they receive |
|---|---|---|
| Experimental: H.P. Achtar Gel 80 U | Experimental | H.P. Acthar Gel (repository corticotropin) Injection, 80 U daily for 10 days, then 80 U twice weekly for up to a total of 48 weeks on therapy. Receives: Drug: Repository Corticotropin Injection |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion of patients with clinically significant improvement - successful glucocorticoid tapering.
Measured: 12 Weeks
Secondary outcomes (1)
Proportion of patients with clinically significant improvement - no need for escalation of other therapy.
Measured: 12 Weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted30 Sep 2016 (estimated)
- Study start1 Feb 2019
- Primary completion2 Nov 2020
- Study completion2 Nov 2020
Why it stopped: Difficulty with recruitment
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for SarcoidosisStudies of the same condition, open and under way.
- More trials run by The Cleveland ClinicThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 4
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 0 participants (actual)
Sponsor and investigators
- Lead sponsor
- The Cleveland Clinic
- Sponsor type
- University, hospital or other
- Collaborators
- Mallinckrodt
- Responsible party
- Sponsor
- Investigators
- Daniel Culver, DO, Principal investigator, The Cleveland Clinic
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02920710
- Sponsor's protocol ID
- ACTHAR CNS
- Status last verified
- Nov 2020
- First submitted
- 29 Sep 2016
- Last updated
- 20 Nov 2020
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 20 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- ACTHAR Therapy for Central Nervous System Sarcoidosis
- ClinicalTrials.gov ID
- NCT02920710
- Acronym / sponsor ID
- ACTHAR CNS
- Conditions
- Sarcoidosis
- Phase
- Phase 4
- Study type
- Interventional
- Recruitment status
- WithdrawnReason stopped: Difficulty with recruitment
- Enrolment
- 0 participants (actual)
- Lead sponsor
- The Cleveland ClinicUniversity, hospital or other
- Collaborators
- Mallinckrodt
- Study start
- 1 Feb 2019 (actual)
- Primary completion
- 2 Nov 2020 (actual)
- Study completion
- 2 Nov 2020 (actual)
- First posted
- 30 Sep 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
There is a need for a more reliable, expeditious therapy that can be used as an alternative to glucocorticoids in severe Central Nervous System (CNS) sarcoidosis. This study aims to provide evidence for effectiveness of ACTHAR gel in CNS sarcoidosis, and provide information about its safety and tolerability
Detailed description
Central nervous system (CNS) involvement is one of the most severe manifestations of sarcoidosis. Sarcoidosis affecting the leptomeninges, spinal cord, or brain parenchyma portends a difficult course and frequently results in severe disability or death (1). Treatment of moderate and severe CNS sarcoidosis typically involves a combination of corticosteroids and cytotoxic agents such as methotrexate (2). Unfortunately, most response rates are reportedly only in the 29-38% range for corticosteroids alone, and the effects of cytotoxic agents in sarcoidosis require up to 6 months to occur. A typical scenario is that patients are treated for prolonged periods with high dose glucocorticoids with suboptimal effectiveness despite development of substantial toxicities. Some series report that cyclophosphamide or infliximab may be beneficial (3), but these approaches are limited by potentially severe toxicities, loss of effectiveness, or payor constraints.
. ACTHAR is a 39-amino acid peptide natural form of adrenocorticotropin hormone (ACTH) that was initially approved in 1952 by the FDA. It has since been approved for 19 indications including respiratory sarcoidosis, multiple sclerosis, and infantile spasms.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Experimental: H.P. Achtar Gel 80 U | Experimental | Drug: Repository Corticotropin InjectionH.P. Acthar Gel (repository corticotropin) Injection, 80 U daily for 10 days, then 80 U twice weekly for up to a total of 48 weeks on therapy. |
Who may qualify
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (8 listed)
- Patient with sarcoidosis as defined by ATS/ERS/WASOG (American Thoracic Society/European Thoracic Society/World Association for Sarcoidosis and Other Granulomatous Disorders)
- Stable baseline immunosuppressive medications
- Moderate to severe disease as defined by at least one of the following criteria:
Key exclusion criteria (7 listed)
- Diagnosis of any underlying neurologic disorder that would potentially confound interpretation of the study results
- Significant change in corticosteroid dose within the past 4 weeks, or other immunosuppressive medication within the past 6 months
- Evidence of current serious infection, or a history of chronic or recurring infections.
- Contraindication to high-dose corticosteroids (e.g. uncontrolled blood sugar).
- Allergies to pig-derived proteins
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Repository Corticotropin InjectionAlso: H.P. Acthar Gel, ACTH Gel, ACTH* Initial treatment with 80 units daily for ten days (induction phase) * Maintenance treatment with 80 units twice weekly (maintenance phase) | Drug | Experimental: H.P. Achtar Gel 80 U |
Primary outcome
Proportion of patients with clinically significant improvement - successful glucocorticoid tapering.
Time frame: 12 Weeks
1 secondary outcome
Proportion of patients with clinically significant improvement - no need for escalation of other therapy.
Time frame: 12 Weeks
Results and safety
The study was withdrawn before enrolling participants, so there are no results.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Nov 2020.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Daniel Culver, DO principal investigator
The Cleveland Clinic
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 4 study testing Repository Corticotropin Injection in people with Sarcoidosis, run by The Cleveland Clinic at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 8 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: withdrawn, as of the sponsor's last update on 20 Nov 2020. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 4 studies take place after a treatment is approved, to learn more about its long-term benefits and risks in everyday use.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study was withdrawn before enrolling participants, so there are no results.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion of patients with clinically significant improvement - successful glucocorticoid tapering. (time frame: 12 Weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Nov 2020.